Amicus Therapeutics Unveils ‘Healing Beyond Disease’ Initiative for Rare Disease Community

Ana de Barros, PhD avatar

by Ana de Barros, PhD |

Share this article:

Share article via email
heart function, exercise capacity

Amicus Therapeutics has launched Healing Beyond Disease, an initiative designed to celebrate people living with rare diseases, like Fabry disease, and their loved ones.

“We are thrilled and honored to officially launch Healing Beyond Disease, our unique promise to further serve the needs of the rare disease community in extraordinary ways. Our core mission at Amicus is to make great medicines,” John F. Crowley, chairman and CEO of Amicus Therapeutics, said in a press release.

Amicus Therapeutics

Credit: Amicus Therapeutics

Healing Beyond Disease was created to enhance the company’s support for patients, families and advocacy organizations in searching for novel medicines. The initiative is based on five pillars and leverages Amicus’ internal resources and expertise.

By bringing time, talent, treasure, a pledge to a cure and building rare bridges, the initiative aims to bring volunteerism, philanthropy and mentorship for individuals, nonprofits and others who are part of the rare disease community.

Healing Beyond Disease, however, is a commitment to do even more than that. It is based on the notion that we are in this business to positively change the lives of people living with rare diseases in many ways. The five unique pillars of our Healing Beyond Disease program reflect our obligations beyond the therapies that we provide. This broader commitment to the rare disease community is a long-term one and it is core to our company beliefs. It includes our philanthropic endeavors, investment in next generation treatments within our current therapeutic areas of focus, and our emerging plans to make our medicines accessible to patients in every part of the world possible — to heal beyond disease,” Crowley said.

“Collaborating with rare disease advocates daily, I believe championing and serving patient communities creates a sense of mission and purpose that inspires all Amicus employees. Healing Beyond Disease is a tangible hallmark of our belief at Amicus that each one of us has the unique ability to do an immense amount of good. This is just the beginning,” said Jayne C. Gershkowitz, chief patient advocate at Amicus.

Amicus is developing its lead candidate compound, migalastat (approved as Galafold in Europe), an oral precision medicine for people living with Fabry disease who have amenable genetic mutations.

Fabry disease is caused by mutations of the GLA gene, which directly affect an enzyme called alpha-galactosidase A (alpha-gal A). Galafold restores the enzyme’s activity in people carrying amenable GLA mutations, who account for 35 to 50 percent of Fabry patients worldwide.